MDA has extended its strategic research partnership with the ALS Therapy Development Institute (ALS TDI) through 2012 and awarded a $2 million grant to support the nonprofit biotech's continued efforts to develop treatments forALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).
The funds were awarded through MDA's ALS initiative Augie's Quest, which focuses on finding treatments and cures for ALS. The $2 million grant brings the total amount Augie's Quest has awarded ALS TDI to more than $26.5 million since 2007.
The extension of the partnership reflects the exceptional progress being made in reaching a common goal: finding therapies for ALS as quickly as possible.
ALS TDI, located in Cambridge, Mass., announced in February 2012 that it will initiate a phase 2 clinical trial in ALS of a drug currently approved for the treatment of multiple sclerosis. The biotech also is testing (in the SOD1 research mouse model) a version of a drug approved for use in humans to treat rheumatoid arthritis. Both drugs target the immune system, which is thought to play a role in the complex ALS disease process.
In December 2011, ALS TDI entered into a research agreement with two major biotechnology companies: Biogen Idec, headquartered in Weston, Mass., and UCB, based in Brussels, Belgium. Upon ALS TDI's completion of testing of another compound that targets the immune system, Biogen Idec and UCB will have the option to license global rights to develop and commercialize the compound as an ALS treatment.
Previous ALS TDI studies conducted in the SOD1 research mouse model have shown that blocking the activation of certain parts of the immune system slows disease progression and improves survival. (For more on the immune system in ALS, see ALS Research: Disconnecting the Immune System.)
Beginning in 2008, the Institute partnered with several MDA clinics nationwide to collect blood and tissue samples from people with ALS and, for comparison, from people without the disease, in order to measure patterns of gene activity (gene "expression").
Also in 2008, ALS TDI published results from an extensive characterization of the SOD1 mouse model of ALS. The findings indicated that a failure to recognize differences among the mice used in studies (such as gender, litter of origin and number of mutated SOD1 genes per cell) could explain why experimental treatments that initially looked good in mouse studies have failed to work in humans. The Institute currently is working to provide a comprehensive characterization of the TDP43 mouse model of ALS.
MDA and ALS TDI forged a historic partnership in January 2007 when they launched a three-year, $36 million collaboration — the largest drug discovery project to date in ALS. (MDA's initial $18 million investment was matched by ALS TDI.)
The project focuses on identifying biological components and pathways in ALS and finding drugs that target them, and a steady rate of progress has proven the collaboration successful.
In 2010, MDA renewed its partnership with ALS TDI with a grant totaling $2.5 million. Based on the biotech's extraordinary progress, the Association awarded a supplemental grant of $855,000 in December of that same year.
The partnership continued in 2011 with another $2 million grant, which helped ALS TDI scientists complete preclinical testing of several different experimental therapies in the SOD1 research mouse model of ALS, and helped expand ALS TDI's research program to include the TDP43 mouse model of the disease. That same year, MDA also awarded a supplemental grant of $1.2 million.